Foundation for Prader-Willi Blog | News

How to Address the Challenges of Developing PWS Therapies

Prader-Willi syndrome (PWS) is a complex disorder, and, to date, supportive care and growth hormone therapy are the only treatments available. The rarity of PWS, the fragmentation of experts and stakeholders involved at each stage of therapeutic development, and limited funding add layers of complexity in the development of safe and efficacious PWS therapies.

Therapeutic development is a risky process that often requires more than 10 years and billions of dollars to complete. Unfortunately, 90 percent of drugs fail due to a lack of effect in patients.

The research plan for PWS needs to be comprehensive and proactive and include the right programs and tools to overcome these challenges and fill existing gaps. FPWR’s grant program and other research initiatives developed over the last decade have greatly advanced our basic understanding of both the genetic and molecular mechanisms of PWS, opening avenues for therapeutic development.

FPWR’s 5-year research plan for PWS comes at a crucial moment to accelerate the development of therapies by acting at each stage of the therapeutic development pathway.

Topics: News, Research

Top 10 Reasons to Come to the FPWR Conference This Year

The FPWR Family Conference takes place August 25-27, 2017, in Indianapolis, IN. Here are the top 10 reasons you need to be here! 

10. Hoosier Hospitality

Our local Conference Host Committee is working behind the scenes to make this the best PWS conference yet! The conference hotel is located in downtown Indywhere you can kayak, pedal boat, bike or relax in a gondola along the scenic Downtown Canal, taking in the sights of public art, war memorials, and the Indy Zoo, Aquarium and Botanical Gardens, located right outside our conference hotel!


Topics: News

2016: Another Remarkable Year for Prader-Willi Research

FPWR community, 2016 was yet another remarkable year for Prader-Willi research! Our supporters have allowed FPWR to fund more research than ever before, taking us closer to treatments for our loved ones with Prader-Willi syndrome. Read below for a few research highlights from the year. 

Topics: News

Announcing our Second Round of PWS Research Grants from 2016!

The Foundation for Prader-Willi Research (FPWR), in collaboration with FPWR-Canada and FPWR-UK, is thrilled to announce our second round of PWS research  recipients for 2016! Eight outstanding projects were selected for funding, totaling $746,200 in support. These projects address a variety of topics important in PWS, including social functioning, hormone regulation, oxytocin and gene regulation. 

Topics: News, Research

Zafgen Donates Hyperphagia Questionnaire to FPWR for Use in Clinical Trials

Zafgen, Inc., has donated its Hyperphagia Questionnaire for Clinical Trials (HQ-CT) instrument to FPWR to help enable the development of potential therapies for Prader-Willi syndrome.

The HQ-CT is derived from a well-established, caregiver-reported questionnaire initially developed by Dr. Elisabeth Dykens and her team at Vanderbilt University that provides an assessment of the food-seeking behaviors common among individuals with PWS, known as hyperphagia. The instrument has been validated in multiple clinical trials and is designed to evaluate a potential drug candidate’s impact on those food-seeking behaviors.

Topics: News

Every Donation Will Be Matched Until Thanksgiving!

We need your help and there has never been a better time to DOUBLE your impact! From now until Thanksgiving, EVERY donation made to our organization will be MATCHED by Leon and Irina Shaulov. 

Topics: News

FPWR Receives $30,000 PCORI Award for Real World Data Workshop

The Patient-Centered Outcomes Research Institute (PCORI) has awarded FPWR $30,000 to support real world data research readiness in the Prader-Willi syndrome community! This support was awarded to FPWR as a Eugene Washington PCORI Engagement Award. 

Topics: News

Elizabeth Roof To Discuss Social Functioning in PWS At Oct. Conference

Individuals with PWS want to be included in social gatherings and are interested in participating in social activities. Often times, however, deficits in social communication and other social skills, combined with the need for food security, can make socializing a challenge, increasing the risk of social exclusion and isolation.

Senior Research Specialist Elizabeth Roof, from the Vanderbilt Kennedy Center, specializes in psychiatric and behavioral development in people with developmental delays. She is well known and revered by many in the PWS community having worked with hundreds of families across the country, both in person and over the phone. 

Topics: News

Genetic Therapy Researchers Work to Reactivate PWS Genes, Identify New Targets

All individuals are born with two copies of chromosome 15: one copy comes from the mother, the other the father. In the case of PWS, the paternal copy is missing a portion of the chromosome (deletion), or is gone altogether(UPD), but the maternal chromosome (in both deletion and UPD subtypes) is present and intact. Through genetic therapy, we may be able to reactivate the maternal chromosome (which is typically silenced) or replace the missing information on the paternal chromosome. 

While genetic therapy is a long-term investment, it has the potential to address the root cause of PWS and possibly reverse the symptoms of PWS. FPWR is currently funding several researchers with projects related to reactivation of the PWS region on the maternal chromosome (that all patients with PWS have). These projects each explore a different approach to the question of whether genetic therapy can be used to effectively treat PWS.

Topics: News

Reiter Covers Differential Gene Expression in PWS at FPWR Conference

To parents, giving away their children's baby teeth can be a big deal! But many parents of children with PWS have gladly answered Dr. Lawrence Reiter's call for baby teeth in the name of researching differential gene expression, as it could lead to successful PWS therapeutics.

Over the past year, Dr. Reiter has been collecting the teeth in order to extract stem cells found in the pulp and transform them into PWS brain cells. FPWR is thrilled to have Dr. Lawrence Reiter presenting at the FPWR Research Conference: Action Changes Tomorrow, October 28-30, 2016.

Topics: News, Research

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